NS-065/NCNP-01 Phase 2 dose finding study
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1 NS-065/NCNP-01 Phase 2 dose finding study PPMD Webinar 1 February 22, 2017
2 Introduction NS Pharma, Inc. (Sponsor) is a wholly-owned, US subsidiary of Nippon Shinyaku Co., Ltd. (Kyoto, Japan) National Center of Neurology and Psychiatry (Tokyo, Japan) is a co-inventor of NS-065/NCNP-01 PharmaLex Development Services, LLC (PDS) is a regulatory affairs and drug development consultancy. CINRG Network and TRiNDS Dr. Paula Clemens is the Phase 2 Study Chair and CINRG Medical Director TRiNDS, overseen by Lauren Morgenroth, is responsible for clinical trial project management with the CINRG clinical sites 2
3 Nippon Shinyaku and NS Pharma Offices Offices Subsidiaries Head Office & Discovery Research Labs. in Kyoto Tajima Shokuhin Kogyo Co., Ltd. Discovery Research Labs. in Tsukuba NS Pharma Inc.(US) Beijing Office Sioe Pharmaceutical Co., Ltd. Odawara Central Factory <Japanese GMP, ISO 14001> London Office 3
4 Study Organization Study Sponsor Regulatory/Strategic Consulting Clinical Project Management CINRG Clinical Study Sites 4
5 Clinical Study: NS-065/NCNP Latest Updates A Phase 2, Dose Finding Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy (DMD) 5
6 Clinical Sites Planned or Possible Anticipated Sites UC Davis Sacramento Soon Washington Univ St. Louis Recruiting Lurie Children s Hospital Chicago Recruiting Children s Healthcare Atlanta Children s Hospital Richmond Soon Duke Univ Durham Univ Florida Gainesville Soon For updated information, please visit at ClinicalTrials.gov 6
7 News Release January 19, 2017 First Patient Dosed in Phase 2 Clinical Study of NS- 065/NCNP-01 for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters, Kyoto City; President, Shigenobu Maekawa) announced today that the first patient dosed in the phase 2 clinical trial of NS-065/NCNP-01 in patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. A phase 2 clinical study in the US and a phase 1/2 study in Japan began recruitment in December 2016 and July 2016, respectively. NS-065/NCNP-01 was registered as "SAKIGAKE designation(japanese version of Breakthrough Therapy Designation) " of the Ministry of Health, Labour and Welfare in Japan in October For more information: 7
8 Phase 2 design Screening Randomization 1 st Muscle biopsy 2 nd Muscle biopsy NS-065/NCNP or 80mg/kg N=6 Placebo N=2 NS-065/NCNP or 80mg/kg N=8 Patient Choice Continue current dose 30-day Follow-up Day-21 Day1 Period 1 (4wks) Week5 Period 2 (20wks) Week24 Extension (24wks) All patients will receive NS-065/NCNP or 80mg/kg Muscle biopsy is conducted twice during the study 80mg/kg cohort will be initiated after safety of 40mg/kg is confirmed (at week5) 8
9 Study Population Approximately 16 patients (4 - <10 years of age) with DMD amenable to exon 53 skipping 9
10 Summary of Inclusion Criteria Patient has a confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 Patient is 4 years and <10 years of age Patient is able to walk independently without assistive devices Patient is able to complete strength and function tests Patient must be on a stable dose of glucocorticoid for at least 3 months prior to study entry 10
11 Summary of Exclusion Criteria Patient has evidence of symptomatic cardiomyopathy Patient has an allergy or hypersensitivity to the study medication Patient is taking any other investigational drug currently or within 3 months prior to the start of study treatment Patient has had surgery within the 3 months prior to the first anticipated administration of study medication Patient has positive test results for HB antigen, HCV antibody or HIV antibody 11
12 Study Assessments Muscle biopsy (Biceps) (at Screening and Week25) Strength and function test (at Screening, Wk13, Wk25) Blood draw 11 visits Assessments Urine collection 11 visits Vital signs Every visit (27 visits) Physical exam 11 visits 12
13 NS-065/NCNP-01 Antisense oligonucleotide with a novel morpholino backbone and molecule size Originated in Nippon Shinyaku jointly with National Center of Neurology and Psychiatry (NCNP) Exon 53 skipping agent (Mechanism of Action) IV infusion, once weekly 13
14 Mechanism of Action Normal Dystrophin mrna DMD Out of frame Normal Abnormal DMD +NS Exon 53 skipping Partly functional protein 14
15 Target Exon of NS-065/NCNP-01 Target Exon NS-065/NCNP-01 is targeted to skip Exon 53 Deleted Exon Patient % out of DMD , 50, 45-50, 48-50, 49-50, 52, , 45-52, 47-52, 48-52, 49-52, 50-52, , 46-48, 46-49, 46-51, , 45, , 44-47, , , 51-53, , 53, Aartsma-Rus, et al. (2009) Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations. Human Mutation 30:
16 Investigator-Initiated Phase 1 Study Design: Open label, Escalating dose, Safety, Efficacy (dystrophin recovery) and PK # of patients: 10 DMD boys Dosing: Once weekly IV infusion for 12 weeks Study site: National Center of Neurology and Psychiatry (1 site) Cohort 1 12 weeks 1.25 mg/kg IV (n=3) Cohort 2 12 weeks 5.0 mg/kg IV (n=3) Cohort 3 12 weeks 20 mg/kg IV (n=4) 16
17 News Release March 23, 2015 An Investigator-Initiated Clinical Trial of NS-065/NCNP- 01 for the Treatment of Duchenne Muscular Dystrophy Has Been Completed Early analysis by NCNP detected dystrophin mrna with the amino acid reading frame restored by exon 53 skipping in every dose group. Furthermore, the expression of dystrophin protein which appeared to have been translated from such a mrna was detected in the high-dose group. On the basis of these results, NS-065/NCNP-01 is expected to have therapeutic efficacy in DMD. In addition, no serious adverse events were observed throughout the study, and no subjects discontinued administration. Anemia and a slight effect on renal function have been reported as general adverse events. For more information: 17
18 Review of several new FDA designations granted to the program IMPORTANT FDA DEVELOPMENTS 18
19 News Release November 09, 2016 FDA Grants Fast Track Designation to NS-065/NCNP-01 for the Treatment of Duchenne Muscular Dystrophy Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters, Kyoto City; President, Shigenobu Maekawa) announced today that the Food and Drug Administration (FDA) has granted Fast Track Designation to NS-065/NCNP-01 for the treatment of Duchenne Muscular Dystrophy (DMD). Fast Track is a process designed to expedite the review of drugs which will be highly effective to treat intractable diseases. NS-065/NCNP-01 is the first antisense oligonucleotide discovered in Japan to be granted Fast Track Designation from the FDA. For more information:
20 News Release January 16, 2017 FDA Grants Orphan Drug Designation to NS-065/NCNP- 01 for the Treatment of Duchenne Muscular Dystrophy Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters, Kyoto City; President, Shigenobu Maekawa) announced today that the Food and Drug Administration (FDA) has granted Orphan Drug Designation to NS-065/ NCNP -01 which is being developed for the treatment of Duchenne Muscular Dystrophy (DMD) in patients who are amenable to exon 53 skipping in the United States (US).The Orphan Drug Designation is issued to drugs which are intended for rare diseases that affect fewer than 200,000 people in the US, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. For more information: 20
21 News Release February 01, 2017 FDA Grants Rare Pediatric Disease Designation to NS- 065/NCNP-01 for the Treatment of Duchenne Muscular Dystrophy Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters, Kyoto City; President, Shigenobu Maekawa) announced today that the Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to NS-065/ NCNP -01 which is being developed for the treatment of Duchenne Muscular Dystrophy (DMD). The Rare Pediatric Disease is defined as a disease that affects fewer than 200,000 persons in the US, primarily aged from birth to 18 years. Under this designation, when an approval is granted for NS-065/NCNP-01, Nippon Shinyaku may be eligible to obtain priority review for a subsequent marketing application for a different product. For more information: 21
22 Reimbursement for Travel Participants in the study will receive reimbursement to support their travel for participation in the clinical trial Greenphire is a company working on behalf of NS Pharma to support this reimbursement process Fast, easy way to make payments Site visit payments by visit description Manual payments Travel Reimbursements Travel Arrangements Opt-in patient messaging strategy 22
23 Patient Advocate For more information, Please contact to Lauren Morgenroth or Visit at ClinicalTrials.gov (NCT ) 23
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